The Amyloidosis Speakers Bureau (ASB), founded in 2019, arranges for ASB patient educators to speak about their diagnostic and treatment experiences with medical students. In 2023, we published a study to understand the impact from the addition of the patient voice to didactic medical education. The study concluded that listening to an ASB patient educator’s narrative was associated with positive attitudes toward communication with patients, interest in acquiring and applying knowledge of amyloidosis, and humility about diagnosis. Post-publishing, continued analysis of the presentation feedback made it clear that another benefit was occurring. During the ASB presentations, questions were repeatedly raised about what guidance the patients might offer to help these budding doctors become better providers and how they could improve their relationships with patients. Their inquiries had nothing to do with amyloidosis and were relevant to every interaction and all diseases. These future providers wanted to be better and wanted the patient’s perspective to help get there. Assessing their questions revealed an unexpected benefit from the patient presentations.
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2024: Mackenzie’s Mission – Our Report Card
This was our seventh full year of operation. We were busy with lots of activities to advance our mission to make a difference in the fight against Amyloidosis. Operationally, we continued to run extremely efficient and lean, and laser-focused on making a difference in multiple ways.
- Raising Awareness about Amyloidosis, which we know can lead to earlier diagnosis, starting treatments sooner, and better outcomes for patients.
- Supporting medical research on Amyloidosis, seeking to understand more, develop better and less invasive diagnostic approaches, and develop more effective treatments, all of which will improve and extend lives.
RAISING AWARENESS
The first part of our mission is raising awareness, for both patients and the medical community.
Amyloidosis Speakers Bureau (ASB)
Back in February 2019 we launched the Amyloidosis Speakers Bureau as the cornerstone of our education effort, focused on closing the medical education gap. It is a direct outreach with live presentations by patient educators, done both virtually and in-person. In addition, each presentation has a clinical discussion about amyloidosis. Thus overall, every presentation has well-rounded and impactful content to educate the audience.
Amyloidosis is considered a rare disease and is not well known. However, there is a belief within the medical community that this disease is not rare, it is underdiagnosed or diagnosed when it is too late to make a difference. The complexity of this disease makes diagnosis one of the biggest challenges affecting patient lives. It is not uncommon to hear from patients that it took multiple years and multiple doctors to ultimately arrive at a correct diagnosis, all the while the disease continues to progress. Until a cure is found, it is imperative to raise awareness within the medical community to close this educational gap so that a diagnosis can be determined much sooner, enabling effective treatments and therapies to slow the disease progression and improve patient survival.
Our response to this crisis is the Amyloidosis Speakers Bureau (ASB), an initiative focused on educating the medical community and closing the education gap about this disease through presentations from amyloidosis patients, a clinical discussion from a medical expert, and our monthly educational updates. Our target audience is future providers (e.g., the next generation of doctors during their first/second year of medical school, and PA students), as well as current medical providers (e.g., internal medicine residency programs for physicians launching their medical career).
During 2024, we gave 86 presentations to over 5,100 future and current medical providers. Since we began in the fall of 2019, we have given over 375 presentations to over 19,500 future and current medical providers! We also are proud to be educating a wide array of specialties who might encounter amyloidosis patients, and every year another medical school cohort graduates – numbering 3,000+, entering the clinical world of practicing medicine and diagnosing patients. Our impact is deepening and continuing to grow.
Amyloidosis Lecture Series
Launched late 2023, we created a lecture series of Expert Insights Into Amyloidosis composed of short educational videos from our ASB Advisors. Developed for our medical student/resident audience, we have found that our patient community is also finding value in these videos. These 10-15 minute videos have thus far proven to be a massive hit. We continue to add to the series at least monthly, and collectively, these videos have amassed over 75,000 views. Shockingly awesome! To view, visit the Education hub of our new website and click on the “Expert Insights” category.
All Things Amyloid podcast
On October 30, 2024 we launched the first dedicated amyloidosis podcast titled “All Things Amyloid.” Found on major podcast platforms, our website, and our YouTube channel, in our episodes we speak with patients and caregivers on a wide array of topics. We also hear from amyloidosis experts about the medical side of this disease. These are short episodes with bi-weekly drops, our initial seven episodes have amassed over 2,100 downloads! To listen to our episodes, visit our podcast website HERE.
Digital Education Initiative
Knowing the critical importance of raising awareness of this disease, it became evident to us that it was a good time to launch a digital initiative to far extend our reach. On June 1, 2024 we launched a targeted educational initiative on our three social media platforms (Facebook, LinkedIn, and X), and a Google search program. Focused topics include cardiac symptoms, west african heritage, extreme fatigue, and carpal tunnel syndrome. After our first six months we have garnered over 14.4 million impressions!
Continuing Medical Student Education: After every presentation we invite students interested in continuing to learn about amyloidosis to join our ASB Briefs mailing list. Today, that list numbers over 1,000! Each month we send a brief discussion about some aspect of the disease with a growing library of links to informative presentations / videos by medical experts, and announcements regarding advancements in treatment. The intention is to keep amyloidosis more front-of-mind and educate on the many facets of this complex and multi-systemic disease.
ADVANCING MEDICAL RESEARCH
The second part of our mission is to help advance research. Research is at the core of developing new therapies to improve patient lives. Patients benefit from research through early access to novel therapies, new tests for earlier/easier diagnosis, and new approved treatments. However, research is expensive, takes many years, and is absolutely critical to the deepening of knowledge fueling these advancements. And while significant progress continues to be made, much more needs to be done.
No progress in the fight against this disease happens without funding, and the NIH provides a mere fraction of what is needed. Researchers require money to run their labs, maintain bio banks, purchase equipment, run clinical trials, and more. To operate, they rely on private foundations (like Mackenzie’s Mission), grants, and individual donors.
Each year, a portion of our budget goes towards advancing research. We are grateful for the support received from donors like you and fundraisers, and proud that collectively these funds are changing the therapeutic landscape and benefiting patients.
In 2024, our donations pushed us over the $1,500,000 threshold for total money donated!
WITH MUCH APPRECIATION AND GRATITUDE
This past year you may have donated cash or securities, sponsored a Facebook fundraiser, supported us during Giving Tuesday, or given us a grant to support our Amyloidosis Speakers Bureau (ASB) medical education initiative. You may have been an ASB patient educator, liked/shared our social media posts, been a guest on or subscribed to our All Things Amyloid podcast, or taken the time to read our posts or newsletter to learn about what’s going on in our community. Whether you did one or many of these, you helped us push forward our fight against this disease and we appreciate your support!
We also want to extend a special thank you to our volunteers (including Linda, Liz, Kathy, Sean, Rayna, Trent, and others!) who passionately and graciously devote their time and expertise. They help our efforts across many aspects of our operations, from management, to speaker development, to research, to graphics/marketing, and video production. Their dedication to our effort is a testament to their belief in what we are doing and we thank them all!
I am encouraged by the impact Mackenzie’s Mission continues to make after just seven short years. Connecting with the amyloidosis community and working together to make an impact is extremely rewarding. There is much work to be done, but with so much help from the community and our supporters I know we can win this fight!
With warm regards for a wonderful and hopeful 2025,
Mackenzie
AN UPDATE ON ME
This past year was a busy one! Mackenzie’s Mission and the Amyloidosis Speakers Bureau accomplished many important projects, laying the foundation for an exciting future. Outside of the foundation, I am now a PA-C, having graduated from the Physician Assistant Program at Northeastern University. Starting March 2025, I will be working as an Inpatient Physician Assistant at Dana-Farber Cancer Institute in Boston. As for my disease, I am monitored closely, and my disease continues to remain under control. I feel great!
ATTRUBY (acoramidis) is now FDA-APPROVED for ATTR-CM in U.S.
Attruby (Acoramidis), was approved by the FDA on November 22, 2024 for ATTR-CM patients (both wild-type and hereditary) in the U.S.
Orally-administered, Attruby is a near complete TTR stabilizer (>= 90%), designed to reduce cardiovascular death and cardiovascular-related hospitalization. In addition, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A.
To honor the courage of our U.S. clinical trial participants, BridgeBio will provide these patients Attruby free for life.
FDA CLEARS AI SCREENING TOOL FOR CARDIAC AMYLOIDOSIS
“Echocardiography is a powerful tool for evaluating cardiac structure and function and is central to the detection and monitoring of disease,” Ross Upton, PhD, CEO and founder of Ultromics, said in a statement. “However, there are some diseases that are very challenging for even the most expert clinician to detect on an echocardiogram. Requiring only a single apical four-chamber image, EchoGo Amyloidosis identifies cardiac amyloidosis and will help drive earlier access to appropriate treatment and care for patients with this underdiagnosed disease.”
Patient Panel Provides Medical Students Insights into Living with a Rare Disease
The Chicago College of Osteopathic Medicine (CCOM) held a patient panel about amyloidosis, a rare disease where abnormal proteins build up in different tissues and organs. The patient panel was an opportunity for medical students to increase their awareness of amyloidosis and hear a patient’s experience living with the rare disease. According to the Amyloidosis Foundation, there are less than 200,000 people in the U.S. who have been diagnosed with amyloidosis.
George Borrelli, D.O., Chair, Clinical Integration, CCOM, welcomed the audience to the patient panel and discussed the importance of the supplemental learning opportunity for the students. “It gives you an opportunity to not only learn about a disease, but to get an account from a patient who has actually gone through the situation.” Dr. Borrelli elaborated, “The earlier this disease is diagnosed, the better the outcomes are. For so many years, this disease has been an enigma, difficult to diagnose, and slipped through many clinicians’ fingers.”
Ozzie Giglio, who is living with amyloidosis, visited Midwestern University and shared his experience with the rare disease. Ozzie is a patient educator with the Amyloidosis Speakers Bureau.
Medical student Kelly Brake (CCOM ’27) said, “It was nice to hear a patient account during our coursework years. It’s a good reminder of the challenges that patients face in healthcare.” She also expressed her gratitude for CCOM’s enrichment of classroom learning with experiences like this event.
The link below is to the full article published by Midwestern University.
Congressional Hill Briefing, Improving Care for Veterans with Rare Diseases: Establishing a National Commission
We, with other members of the amyloidosis community, were proud to join an important meeting hosted by the Center for Patient Advocacy Leaders (CPALs). They hosted a Congressional Hill Briefing, Improving Care for Veterans with Rare Diseases: Establishing a National Commission, at the U.S. Capitol Visitor Center in Washington, DC. This briefing was designed to bring together Veterans/Veterans’ advocates, rare disease advocates, patients, and congressional staff to address unmet needs of Veterans with rare diseases and explore collective action to help ensure Veterans with rare diseases get the comprehensive, patient-centered care and treatment they need and deserve.
Mackenzie’s Mission recognized by Mayo Clinic in the Hall of Benefactors
We thank the Mayo Clinic for the recognition of our ongoing support of their Amyloidosis Research Fund. Starting in 2017, shortly after Mackenzie’s Mission was founded, our cumulative support has been meaningful, and we are now Distinguished Benefactors.
In addition, we are proud to be in the Mayo Clinic Hall of Benefactors!
Dr. Morie Gertz at the Mayo Clinic works with a multidisciplinary team of experts and helps to lead research initiatives which are widely recognized worldwide as one of the original amyloidosis centers of excellence.
While we know that funding research, bio banks, conducting clinical trials, and more requires significant funds, it is our hope that our year-to-year support helps them do more and make an even bigger difference.
We are eternally grateful to the amyloidosis team at the Mayo Clinic and all they do for our patient community to improve lives.
Mackenzie’s Mission at the Bradley Z Naifeh Amyloidosis Conference 2024!
We were proud to be part of the 2nd annual Bradley Z. Naifeh Amyloidosis Conference 2024 at Houston Methodist! On day 1 the auditorium was filled with healthcare professionals who learned about the many facets of the clinical side of amyloidosis from experts such as Dr. Ron Witteles of Stanford and Dr. Angela Dispenzieri of Mayo Clinic.
On day 2 the auditorium was packed with patients and caregivers of all types of amyloidosis, offering a wonderful opportunity to learn about the disease, resources available, and network with other patients and caregivers.
Mackenzie and Deb had the chance for a photo op with Megan Fleischfresser Naifeh (daughter of Bradley Z. Naifeh who lost his battle with AL Amyloidosis) and Dr. Arvind Bhimaraj (co-host of the conference).
We thank the Naifeh family for their support of this valuable annual conference.
ASB Participates in Rare Disease Day 2024!
The Amyloidosis Speakers Bureau was thrilled to participate on Rare Disease Day! One way we were part of the celebration was for our patient educator Sean to share his journey at Keck Graduate Institute’s Rare Disease Awareness Fair.
The Story Behind the Amyloidosis Speakers Bureau
The story behind the Amyloidosis Speakers Bureau (ASB) is very special. In this video we hear where the original concept of patients presenting to medical students came from — Dr. Gordon Huggins of Tufts University School of Medicine. Hear how he was “auditioning” his patients to speak to his class of second year cardiovascular medical students, and then he met Charolotte Raymond. It was an experience so meaningful it inspired Charolotte to conceptualize a program whereby patients would educate medical students across the country. She partnered with Mackenzie’s Mission in 2018, where together we took her original concept and collectively developed it into the Amyloidosis Speakers Bureau. The ASB was officially founded February 1, 2019.
